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The statistics of phase 0 trials

  1. Author:
    Rubinstein, L. V.
    Steinberg, S. M.
    Kummar, S.
    Kinders, R.
    Parchment, R. E.
    Murgo, A. J.
    Tomaszewski, J. E.
    Doroshow, J. H.

  2. Author Address

    [Rubinstein, Larry V.; Tomaszewski, Joseph E.; Doroshow, James H.] NCI, Div Canc Treatment & Diag, Bethesda, MD 20892 USA. [Steinberg, Seth M.; Kummar, Shivaani; Doroshow, James H.] NCI, Ctr Canc Res, Bethesda, MD 20892 USA. [Kinders, Robert; Parchment, Ralph E.] NCI, Lab Human Toxicol & Pharmacol, Appl Dev Res Support Directorate, SAIC Frederick Inc, Frederick, MD 21702 USA. [Murgo, Anthony J.] US FDA, Off Oncol Drug Prod, Silver Spring, MD USA.;Rubinstein, LV, NCI, Div Canc Treatment & Diag, Bethesda, MD 20892 USA.;rubinsteinl@ctep.nci.nih.gov
    1. Year: 2010
    2. Date: May
  2. Journal: Statistics in Medicine
    1. 29
    2. 10
    3. Pages: 1072-1076
  4. Type of Article: Proceedings Paper
  5. ISSN: 0277-6715
  1. Abstract:

    The PD-driven phase 0 trial is a new form, designed to be a first-in-man study, often of a new agent, conducted to assess drug effect on a molecular target, by means of a pharmacodynamic (PD) assay, in a very small number (10-15) of patients. Such a study is meant to be a proof of principle trial to determine whether the agent yields the PD effect predicted by pre-clinical studies. The dosage is meant to be pharmacologically active, but is neither toxic nor likely to yield clinical benefit. Such a trial may be used to serve as a very early test of an agent's biologic effect, allowing for early weeding out of ineffective agents, or as an early means of determining the most promising of competing analogue agents. This manuscript will present designs for such PD-driven studies that are statistically efficient and rigorous, focusing on non-comparative trials. The phase 0 trial promises to become an increasingly important tool for facilitating and speeding the development of new therapeutic agents, particularly in oncology. Copyright (C) 2010 John Wiley & Sons, Ltd.

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External Sources

  1. View Full Text
  2. DOI: 10.1002/sim.3840
  3. View record in Web of Science®
  4. WOS: 000277735900003

Library Notes

  1. Fiscal Year: FY2009-2010
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